VERMILLION Diagnostic Bioinformatic Solutions to Change Gynecologic Outcomes NASDAQ: VRML NOVEMBER 2017
FORWARD-LOOKING STATEMENTS This presentation contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995. Words such as may, expects, intends, anticipates, believes, estimates, plans, seeks, could, should, continue, will, potential, projects and similar expressions are intended to identify such forward-looking statements. Readers are cautioned that these forward-looking statements speak only as of the date of this presentation, and the Company does not assume any obligation to update, amend or clarify them to reflect events, new information or circumstances after such date except as required by law. Company estimates set forth in this presentation are based on various sources of information and various assumptions and judgments made by the Company, which Company management believes are reasonable. However, the Company cannot assure you that Company estimates are correct, and actual data may materially differ from Company estimates. The forward-looking statements reflect the views of the Company as of the date of this presentation and are subject to certain risks, uncertainties and assumptions, including those described in the section entitled Risk Factors in the Company s Annual Report on Form 10-K forthe year ended December 31, 2016 and quarterly report on Form 10-Q for the quarter ended March 31, 2017. This presentation is copyright 2017 by Vermillion, Inc. All Rights Reserved. 2
COMPANY OVERVIEW Ticker Symbol: VRML (NASDAQ CM) Market Cap (11-03-2017): $104.4M Shares Outstanding (10-31-2017): 60.0M Cash and Cash Equivalents (09-30-2017): $7.8M Enterprise Value: $98.3M 3
VERMILLION S FOCUS ON WOMEN'S HEALTH OUR VISION To be the global diagnostic platform leader in advancing women s health with information and technologies to improve outcomes OUR MISSION To allow women with pelvic pain and masses to have painless and disease-free lives COLLABORATIONS With leading scientific and clinical institutions such as John Hopkins, UCI, MD Anderson, Moffitt, Kaiser, DOD and Quest Diagnostics FLAGSHIP PRODUCT/ GLOBAL Dx PLATFORM OVA1, the first FDA-cleared, multi-biomarker liquid biopsy for pre-surgical triage of ovarian cancer >100,000 performed to date 4
OUR ORGANIZATION Vermillion, Inc. (Nasdaq: VRML) is dedicated to the discovery, development and commercialization of bio-analytical solutions that help physicians diagnose, treat, and improve gynecologic health outcomes for women. ASPiRA LABs is a wholly owned specialized women s health reference laboratory of Vermillion, Inc. ASPiRA IVD provides custom solutions for IVD clinical trials and studies for complex diagnostics. OVA1 was the first FDA-cleared Multivariate Index Assay (MIA) to measure the risk of ovarian cancer malignancy. Overa is the second generation Multivariate Index Assay (MIA2) to measure the risk of malignancy. Our vision is to be the global leader in personalized medical management of gynecologic health to improve clinical outcomes for patients, physicians and payers. 5
INVESTMENT HIGHLIGHTS Target market expanding to > $1 billion with the OVA family of products* Key investor support from Jack W. Schuler and Oracle Investment Management 2016 2017 March: 510(k) marketing clearance from the FDA for Overa April: Published first significant clinical utility data for OVA1 June: Recorded initial revenue from ASPiRA IVD operations July: Payer contracts with Priority Health Managed Benefits, Independent Medical Systems and Sutter Valley Medical Foundation (d/b/a Gould Medical Foundation) September: Signed payer contract with CareFirst BlueCross BlueShield October: Launched Pelvic Mass Specimen and Data Repository October: Overa launched in the U.S. via Targeted Launch Program November: OVA1 receives Level B Recommendation in the ACOG Clinical Management Guidelines for the Management of AdnexalMasses December: Received a Proprietary Laboratory Analyses (PLA) code (0003U) for Overa (MIA2G) from the American Medical Association February: Published Significant Study Combining OVA1 and Symptomatology which resultedin 100% Sensitivity and Negative Predictive Value March: Announced in-network agreement for TriCare South, 2.5 million lives Q1/Q2: IVD services landed 2 top pharmaceutical trial service agreements including one enrollment study July: Expanded patient advocacy program nationally to assist patients with proactive benefit checks July: Foundational OVA1 health economics manuscript accepted for publication September: Preliminary PAMA pricing published for OVA1 and Overa October: Payer contracts totaling 9 million lives. Major ones include National Tricare and BCBS Louisiana November: Positive medical policy expanded for 14 providers totaling over 26 million lives including Health Care Service Corporation (HCSC), Horizon BCBS, Highmark BCBS, and Wellcare *Company estimates 6
VERMILLION HAS A LARGE MARKET OPPORTUNITY U.S. healthcare system annually spends 1 $22B on endometriosis care, $5.1B on OvCa 2 3 care, and $1.2B on PCOS care 1. UCSF School of Medicine 2. National Cancer Institute 3. Jason, et al. JAMA Internal Medicine 171(13) 4.American Cancer Society 5. Company estimates 6. www.womenshealth.gov 15K 4 22K 4 100K-300K 75-90% benign 5 5 500K-1M 98% benign Physiologic cyst Benign mass Cancer Non-Gyn mass 5 5 20M Confusion Matrix of Diagnosis Endometriosis; PCOS; Functional Cysts 6 Ovarian Cancer Deaths Ovarian Cancer Pelvic Masses to Surgery Pelvic Masses Pelvic Mass Diseases Product Development 2010-2016 2017-2020 OVA1 OVERA OVA1+ OVA1 2.O DxA1 DxA2 7
OVARIAN CANCER: THE SIZE OF THE PROBLEM Ovarian cancer stage at presentation and survival Description Incidence 5-Year Survival Localized 15% 93% Regional 20% 73% Distant 60% 29% Unstaged 6% 25% 4 >60% of ovarian cancers are diagnosed at late stages 5 5 6 Cancer Site Ovarian Breast 5-Year Mortality Rate 55% 11% SEER data Adapted from Barakat RR, et al. Principles and Practice of Gynecologic Oncology, 5th ed. Baltimore, MD: Lippincott Williams & Wilkins, 2009 8
DESPITE THIS UNMET CLINICAL NEED no solution exists. Current standard of care is inadequate to manage patients and results in poor outcomes and increased costs 5 6 1. Current clinical assessment is inadequate Physical exam is specialist-dependent Imaging is subjective 5 4 2. CA-125 is used off-label 1 /Alternative Technology ROMA Both have high rate of false negatives 2,3 CA-125 is non-specific; elevated in endometriosis, liver, GI, breast, etc. 4 3. Preoperative biopsy is medically inappropriate 1. Moss ET, et al., Clin Pathol 2005;58:308-312; 2. Bristow RE, et al., Gynecol Oncol. 2013;128:252-259; 3. Lennox G, et. al., Int l J Gynecol Cancer. 2015; 809-14, Vol 25; 4. Fritsche HA, and Bast RC, Clin Chem. 1998 Jul;44(7):1379-80 9
VERMILLION S SOLUTION 1 ST GENERATION MIA (Multi-Variate Index Assay) November 2016, ACOG Guidelines FDA-cleared liquid biopsy blood test, which pre-operatively assesses risk of ovarian cancer and helps triage high-risk patients The OvaCalc algorithm five biomarker results into a single numerical test value related to the probability of malignancy Interpret OVA1 on a 0-10scale B2 Microglobulin Host response CA-125II Released by tumor cells <5.0 5.0 Pre-menopausal 0 1 2 3 4 5 6 7 8 9 10 Cholesterol transport Apolipoprotein A1 Hormone and vitamin transport Prealbumin Iron transport Transferrin Low probability of ovarian cancer <4.4 4.4 Post-menopausal High probability of ovarian malignancy 0 1 2 3 4 5 6 7 8 9 10 Low probability of ovarian cancer High probability of ovarian malignancy 10
VERMILLION S SOLUTION 2 nd GENERATION MIA2G FDA-cleared liquid biopsy blood test, which pre-operatively assesses risk of ovarian cancer and helps triage high-risk patients The OvaCalc algorithm five biomarker results into a single numerical test value related to the probability of malignancy FDACleared Mar 2016 Launched Q4 2016 New Reimbursement Code Dec2016 New Preliminary PAMA Price Sept 2017 Protein Function Apolipoprotein A1 Cholesterol transport HE4 (Human Epididymis Protein 4) Host response CA-125II Released by tumor cells Transferrin Iron transport Hormonal Status FSH (Follice-Stimulating Hormone) Menopausal State 5.0 OVERA TEST RESULT: One Cutoff for All Ages 0 1 2 3 4 5 6 7 8 9 1 0 LOW RISK HIGH RISK 11
KEY PUBLICATIONS TITLE Author, Institution N Conclusions Effectiveness of a multivariate index assay in the preoperative assessment of ovarian tumors Ueland, et al. University of Kentucky 524 Patients First pivotal trial of OVA1, n=590 OVA1 detected 76% of malignancies missed by CA125 The performance of OVA1 was consistent in early and late-stage cancers Ovarian malignancy risk stratification of the adnexal mass using a multivariate index assay Bristow, et. al. UC, Irvine 494 Patients Second pivotal trial of OVA1 with 494 surgeries OVA1 added to clinical impression (CI) reduced cancers missed from 26% to 4% NPV was 98%, PPV was 31% Clinical performance of a multivariate index assay for detecting early-stage ovarian cancer Longoria, et al. UC Irvine 1016 Patients Compared CA125, CI or mod-acog standard of care to OVA1 in early-stage OvCa OVA1 with CI reduced early-stage cancers missed from 31% to just 5% The effect of ovarian imaging on the clinical interpretation of multivariate index assay Goodrich, et al. University of Kentucky 1024 Patients Compared OVA1 head to head or in combination with ultrasound or CT scan US missed 23% of OvCa, CT missed 20% Using OVA1 with US reduced OvCa missed to just 2% Study suggested that OvCa risk can be personalized by a multi-modality algorithm Validation of a second-generation multivariate index assay for malignancy risk of adnexal masses Coleman, et al. 493 Patients Overa s sensitivity & NPV higher than CA125 or mod-acog industry benchmarks Specificity (69%) and PPV (40%) improved over OVA1 (54% & 31%, respectively) Same high-risk cutoff for all women, unlike CA125, mod-acog, ROMA or OVA1 12
BEST CARE PATHWAY LEVEL A AND B, ACOG 13
NEW OVA1Plus REPORT Level A (TVUS) + Level B (OVA1) (MIA) on One Report The new OVA1 Report now includes Ultrasound 1 Risk to assess risk of malignancy with 2 modalities (TVUS + OVA1, ACOG Level A and B ) Addition of CA-125, for baseline for newly diagnosed OvCA patients 1. Goodrich ST, et. Al. Am J Obstet Gynecol. 2014 Jul; 211(1): 65e1-65e11 14
OVA1 VS. STANDARD OF CARE STAGE I AND II 85% Reduction 100% 50% 37% 63% 31% 69% 23% 77% 8% 92% 5% 95% Rate ofcancer MISSED Rate of Cancer DETECTED 0% CA-125 II* CA-125 II + Clinical Assessment Modified ACOG** OVA1 OVA1 + Clinical Assessment (n=1016surgeries, with 86 early stagecases, 61 StageI, 25 Stage II) Clinical Impression included physician examination and imaging, per the study inclusion criteria, and CA-125,if used *Significant difference in sensitivity as compared to OVA1 + Clinical Assessment (from McNemar s test p<0.05) ** High risk pre-menopausal: CA-125 (>67 U/mL), ascites, or evidence of abdominal/distantmetastasis. Postmenopausalwomen: CA-125(>35U/mL), nodular or fixed pelvic mass, ascites, or evidence of abdominal/distantmetastasis. 1.Longoria TC, et al. 2014Clinical performance of a multivariate index assayfor detecting early-stagecancer. Am J Obstet Gynecol doi: 10.1016/j.ajog.2013.09.017. 15
OVA1/OVERA VS. STANDARD OF CARE 16
PUBLICATIONS DRIVE STANDARD OF CARE/REIMBURSEMENT Published Evidence Ueland, et al Obstetrics and Gynecology, 2011 Bristow, et al. Gynecologic Oncology, 2013 Am J Gynecol, 2013 Longoria, et al. Am J Obstet Gynecol, 2013 Goodrich, et al. Am J Obstet Gynecol, 2015 Forde, et al. Curr Med Res Opin, 2015 Coleman, et al. Am J Obstet Gynecol, 2016 Eskander, et al. Am J Obstet Gynecol, 2016 Guidelines/Position Statement ACOG PracticeBulletin Number 174, November 2016, page 10 National Comprehensive Cancer Network (NCCN) Guidelines, Version 1, 2016 Updated Aug. 26, 2016 Positive Policy Coverage/Contract Positive Policy Coverage/Contract 17
INTELLECTUAL PROPERTY Granted Pending Family USA Ex US Total USA Ex US Total 18 63 81 10 34 44 25 Issued patents covering various ovarian cancer biomarkers Pending patent applications covering OVA1 and Overa products Algorithm kept as trade secret 18
Standard Risk Stratification OVERA BREAKTHROUGH IN OVARIAN CANCER MANAGEMENT Sensitivity Specificity PPV NPV OVA1 1 (95%CI) 92% (85.1-96.3) 54% (48.6-58.3) 31% (26.0-37.0) 97% (93.6-98.5) Veracyte 2 Afirma GEC 92% 52% 32-47% 93-96% Overa (95% CI) 92% 69% 40% 97% Overa performance exceeds OVA1 performance statistically 1.Bristow RE, et al., Gynecol Oncol. 203:128:252-259 2.Alexander EK, et al., N Engl J Med 2012 Aug 23; 367(8):705-15 2015 ASCO poster on OVA2 validation available upon request as PDF or hard copy Overa performance meets or exceeds OVA1 Overa performance lower than OVA1, but not statistically different 19
WE SAVE MORE LIVES AND REDUCE MORE COST BY EVOLVING OUR STRATEGY WITH OVA1 AND OVERA To improve patient care, we plan to expand our scope by classifying all pelvic mass patients to determine the best disease management care path 500K to 1M pelvic masses diagnosed 1 100-300K 1 400-700K 1 Correct referral for ovarian cancer surgery Correct treatment pathway Benign Gynecologic Disease Malignant Gynecologic Disease; Non-Ovarian Diagnostic Applications Surgery vs. watchful waiting, best surgical approach Recurrence and monitoring Improved malignancy detection ID of other pelvic mass disease 2M CA-125 Tests in U.S. 1. Companyestimates 20
BUILDING ON OUR BIG DATA FOUNDATION To impact this expanded scope, biomarkers alone are limited in terms of needed algorithm development and accuracy Unparalleled Specimen and Data Repository Clinical Data Imaging/ Other Modalities Proteomic/Nucleic Acid Markers FOUNDATION FDA cleared algorithms IP FDA/LDT Expertise Algorithm Platform PROOF OF CONCEPT LAUNCHED OVA1 PLUS TVUS November 2016 Impact more than 1M patients 1 A platform that can be commercially replicated 21
PRODUCT PIPELINE 22
EXPANDED OPPORTUNITY SIZE US + EX US US>1M TAM EX US > 10M TAM Differential Dx of pelvic disease US 250K-1M TAM EX US 2.5M-10M TAM Rule out; watchful waiting, surgical choice, disease progression, recurrence monitoring US100-300K TAM EX US 1M-3M TAM Guided referral OVERALL TOTAL $2.2B - $4.4B US $1.2B-$2.4B EX US $1.0B-2.0B US $440M $880M ES US $500M-$1.0B US $67M-$200M EX US $100M-$300M 23
THE VALUE OF VERMILLION S SOLUTIONS FOR ALL CUSTOMERS To Patients Improve time to Tx Low risk: peace of mind High risk: get to the right specialist To Providers To Payers Improve differential Dx Low risk: keep patient High risk: refer to Gyn Onc Increased control of quality outcomes in ACA climate Reduction in unnecessary procedures Improved health economics demonstrates significant QALY Right Tx /right patient /right cost 24
COMMERCIALIZATION STRATEGY Payers Payer strategy to increase utilization and drive price based on HECON Influence Gyn Oncs to drive volume in referral base Gyn Onc OB/Gyn 1:38 ASPiRA Labs (Testing and patient data) Hospital Systems/ Regional Labs KOL strategy in hospital systems and regional lab contracts to drive care pathways 25
OVA1 COVERAGE YTD PATIENT LIVES IN MILLIONS Goal: Targeted Growth with Positive Medical Policy Decisions 26
CLINICAL UTILITY PUBLICATION Purpose: To assess OVA1 s ability to drive referral of ovarian cancer patients to GYN ONC prior to their first surgical intervention Method: Retrospective standardized survey of OVA1 prescribers to report on ovarian cancer cases where OVA1 was prescribed 22 physicians with 5 OVA1 results 136 OVA1-Elevated risk scores 122 Surgeries resulted fromthese 42 benign tumors 2 non-ovarian malignancies 65 primary ovarian cancers 10 of these LMP 31 of these early stage (48%) Conclusion: High-risk OVA1 was associated with 94% Gyn Onc surgery and 100% consultation if primary OvCa, demonstrating clinical utility of the test *Current Medical Research and Opinion, 2016, Vol. 32. No. 6, 1161-1165 http://dx.doior/10.1080/03007995.2016.1176014, ArticleFT-0129.R1-0000.XO/1176014 All rights reserved: reproduction in whole or part not permitted 27
COST EFFECTIVENESS MODEL ANALYSIS Assay Lab List Price ICER OVA1* ASPiRA $1,495 $12,189/QALY 1 OVA1** ASPiRA $1,495 $35,094/QALY 1 BrCa 1/2 Test Myriad Genetics $2,436 $36,845/QALY 2 Oncotype DX Genomic Health $4,175 $15,578/QALY 3 Considered cost effective when under threshold $50,000/QALY Use of OVA1 resulted in a fewer projected re-operations and pre-treatment CT scans versus CA-125II (69%-fewer) or macog (51% fewer) OVA1 increased the quality adjusted life years(qaly) of the patient cohort OVA1 was shown to be cost effective versus macog ($35,094/QALY) or CA-125($12,189/QALY) as both were below the $50,000/ QALY ICER threshold Incremental cost-effectiveness ratio (ICER): Average incremental cost associated with one additional unit of the measure of effect (i.e., QALY) between two possible interventions Quality-adjusted life-year (QALY): A measure of quality of life that takes into account both the quantity and the quality of the life generated by interventions *Compared to CA-125II **Compared to modified ACOG guidelines 1. Forde GK, et al., Curr Med Res Opin. 2015Dec 7:19 2. 3. Li Q, et al., San Antonio Breast Cancer Symposium 2011,San Antonio, TX Vanderlaan BF, et al., 2011.Am J Manage Care. 2011;12(7):455-464 28
BUDGET IMPACT MODEL: ECONOMICS OF OVA1 USED AS AN EFFECTIVE ASSESSMENT TOOL IN THE TREATMENT OF OVARIAN CANCER Supports the use of OVA1 instead of CA125 by indicating that cost-savings can be achieved OVA1 also supports clinical benefits of improved diagnostic accuracy, early disease detection, and reductions in multiple, and possibly unnecessary, referrals to gynecologic oncologists CA125 and OVA1 Included OVA1 Penetration Rate Commercial Medicare 20% $.06 $.01 25% $.07 $.01 30% $.09 $.02 35% $.11 $.02 40% $.13 $.03 45% $.15 $.04 50% $.17 $.05 Sensitivity analysis revealed potential savings of up to $0.17 PMPM for commercially insured patients and up to $0.05 for Medicare beneficiaries Benchmark: GHI BIM = $0.03-$0.05 PMPM CA125 and OVA1 Excluded Commercial Medicare $.08 $.02 $.10 $.03 $.12 $.04 $.15 $.05 $.17 $.06 $.20 $.07 $.23 $.08 This table illustrates the combined (i.e. low and high risk) cost impact based on various levels of OVA1 penetration in the patient populations, including and excluding the cost of OVA1 and CA125. Brodsky, B. S., Gary, G. M., Scotti, D. J., Needham, K. A., Cool, C. L. (2017). Economic Impact of Increased Utilization of Multivariate Assay Testing to Guide the Treatment of Ovarian Cancer: Implications for payers. American Health & Drug Benefits, 10(7). Retrieved from http://www.ahdbonline.com/issues/2017/october-2017-vol-10-no- 7/2492-economic-impact-of-increased-utilization-of-multivariate-assay-testing-to-guide-the-treatment-of-ovarian-cancer-implications-for-payers.
EXPERIENCED MANAGEMENT TEAM IN PLACE 31
CURRENT FINANCIAL PRIORITIES (Value Based Price Starting in 2018) 32
VERMILLION S ANTICIPATED MILESTONES Overa Cleared validation OVA1 HECON Overa Direct to Consumer/DTW Launched Mar 2016 OVA1 +1.0 Launched Nov 2016 ACOG Guidelines OVA1/Overa OVA1/Overa plus 2.0 Symptom OVA1/Overa clinical utility/hecon OVA1/Overa plus 1.0 plus 2.0 Pama Price Jan 2018 Volume multipliers + Sales/ Regional lab strategy DxA I DxA I Validation studies DxA II DxA I HECON studies Key Catalysts: Publications o Clinical Utility/Health Econ National Payer National Distribution Partner(s) 2015 2016 2017 2018 2019 2020 Gain positive Medical Policy at major health plans (25M) Increase in contracting for OVA1/Overa ~80% coverage Medical Policy for OVA x auto add to existing contracts Medical Policy for DxA auto add to existing contracts 33
SUSTAINABLE ADVANTAGES, TAILWINDS AND HEADWINDS Sustainable Advantages Biomarker IP Trade secret algorithm ASPiRA Labs data structure and source Tailwinds FDA and LDT process experience Robust clinical/health economics publication pipeline World-class clinical partners Headwinds Payer contractingdriving rigorous plan forward Test adoption/habit Cumbersome guideline process DONE ACOG Adnexal Mass NCCN Ovarian Cancer SGO Position Statement To Complete ACOG Ovarian Cancer 34
VERMILLION S STRATEGY: BUILT FOR THE FUTURE Strategic Strengths and Capabilities Myriad Gx Health Exact Sci VRML FDA-cleared product Unique Category 1 CPT code (AMA) Medicare Coverage/Gap Fill Pricing IVD Kit CLIA lab for LDTs Multi-modal predictive analysis Proprietary disease registry/samples Leading peer-reviewed publications Strong outreach: KOLs/advocacy 35
CONCLUSIONS First of its kind, FDA-cleared multi-variate global technology platform In two pivotal trials, OVA1 detected >80% cancers missed by CA-125 and physician assessment 1,2 2016-17 Progress March 2016-2 nd Generation, Overa, FDA cleared April 2016, 1 st Clinical Utility July 2016- March 2017, Payer Progress November 2016, ACOG Guideline Inclusion March 2017, Announced in-network agreement for TriCare South Q1/Q2 17: IVD services landed 2 top pharmaceutical trial service agreements including one enrollment study July 2017: Foundational OVA1 health economics manuscript accepted for publication September 2017: Preliminary value-based PAMA pricing published for OVA1 and Overa October/November 2017: Positive medical policy expanded for 14 providers totaling over 35 million lives including plans associated with Health Care Service Corporation (HCSC), National Tricare, Horizon BCBS, and Highmark BCBS Risks mitigated Strong barriers to entry Market leader expertise: data and specimen bank, biomarkers, algorithm development and clinical/data partnering to develop solutions Pending patent applications OVA1 and Overa, various ovarian cancer biomarkers 1.Bristow RE, et al., Gynecol Oncol. 2013;128:252-259 2.Ueland FR, et al., Obstet Gynecol. 2011 June; 117(6):1289-97 36
Corporate Headquarters 12117 Bee Caves Road Building III, Suite 100 Austin, TX 78738 Tel: (512) 519-0400 Fax: (512) 439-2038 www.vermillion.com ASPiRA IVD 35 Nutmeg Drive Suite 260 Trumbull, CT 06611 Tel: (203) 993-8300 Fax: (203) 502-8680 Investor Relations Michael Wood LifeSci Advisors LLC. Tel: (646) 597-6983 mwood@lifesciadvisors.com